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Research

Understanding Amyotrophic Lateral Sclerosis

Amyotrophic lateral sclerosis (ALS) is a degenerative motor neuron disease typically diagnosed in adults in middle age or later. About 18,000 people in the United States are currently living with ALS, making it one of the more common neuromuscular diseases, and yet there is still a lot for scientists to learn about why it occurs and how to treat it.

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A Successful In-Person Conference

In mid-March, more than 1,000 people gathered in Nashville, Tennessee, for MDA's 2022 Clinical & Scientific Conference."It's a delight to meet again in person," said MDA President and CEO Don Wood, PhD, in a welcome address. "For many of you, it's the first time on a plane in two years."

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A New Direction for Charcot-Marie-Tooth Disease

Charcot-Marie-Tooth disease (CMT) is not one disease but a spectrum of nerve disorders. It is named after the three physicians who first described it in 1886: Jean-Martin Charcot and Pierre Marie of France and Howard Henry Tooth of the United Kingdom. CMT affects the peripheral nerves that run through the arms and legs, connecting the brain and spinal cord to the muscles and sensory organs. It occurs in approximately 1 in 2,500 people.

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Family Inheritance

In genetics, inheritance doesn't refer to property or financial assets. It is the process by which genetic information is passed from parents to children. Most neuromuscular diseases are genetic disorders, meaning they are caused by changes in our genes. While these changes, called gene mutations or variants, often are inherited from parents, they don't always cause disease. For many neuromuscular diseases — and the number grows every year with continuing research — scientists have identified disease-causing gene variants. They also have been studying the inheritance patterns, meaning how a gene variant may be passed from one generation to another and how it might affect members of a family.

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The Latest on Mitochondrial DNA Depletion/Deletion Syndrome

Mitochondrial DNA (mtDNA) depletion/deletion syndrome (MDDS) is a group of genetic diseases in which the mtDNA inside cells cannot replicate correctly. Mitochondria are the powerhouses of cells, generating more than 90% of the energy in our cells. With fewer functioning mtDNA, cells fail to generate enough energy to function properly.

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Demystifying Drug Prices

Back in December 2016, when the US Food and Drug Administration (FDA) approved Spinraza — an intravenous therapy developed by Biogen to treat all types of spinal muscular atrophy (SMA) — parents, patient advocates, and even industry observers were shocked at its price: $750,000 for the first year, then $375,000 every year after, for the rest of a patient's life.

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Researchers Want to Know More About Oculopharyngeal Muscular Dystrophy

Oculopharyngeal muscular dystrophy (OPMD) is rare, even among muscular dystrophies, with an incidence of 1 in 100,000 people. It is a late-onset disease, with symptoms typically first appearing after age 50.OPMD gets its cumbersome name from “oculopharyngeal,” meaning that the disease affects the muscles of the upper eyelids and the throat. People with OPMD generally develop droopy eyelids, called ptosis, as well as difficulty swallowing, or dysphagia. They also can experience progressive weakness in the legs.

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One in a Million

All the neuromuscular diseases MDA covers are considered rare, meaning they affect fewer than 1 in 20,000 individuals. But within each of those diseases, there are dozens of subtypes defined by the genetic mutations that cause them. Many, if not most, are considered ultra-rare, affecting fewer than 1 in 50,000.

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How to Make a Decision About a Clinical Trial

Colleen and Chris Labbadia of Geneva, Fla., became concerned when their son, Brendan, was slow meeting early physical milestones. He was diagnosed with Duchenne muscular dystrophy (DMD) before his second birthday, and when he was 4, they enrolled him in a clinical trial for an experimental drug. "It was the hope for a better future for Brendan," Colleen says.

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Express Delivery

Usually, viruses are bad news. But lately, they’re getting good press, thanks to the COVID-19 vaccines created by AstraZeneca, Johnson & Johnson, and Immunity Bio, which use viral vectors to deliver instructions to our cells to fight off the novel coronavirus.Delivering immunity isn’t the only thing viral vectors can do. They also hold great promise in gene-targeted therapies for neuromuscular diseases.

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